EP3758724A4 - Nouveaux vecteurs de virus adéno-associés (vaa), vecteurs de vaa présentant une désamidation de capside réduite et utilisations associées - Google Patents

Nouveaux vecteurs de virus adéno-associés (vaa), vecteurs de vaa présentant une désamidation de capside réduite et utilisations associées Download PDF

Info

Publication number
EP3758724A4
EP3758724A4 EP19760264.2A EP19760264A EP3758724A4 EP 3758724 A4 EP3758724 A4 EP 3758724A4 EP 19760264 A EP19760264 A EP 19760264A EP 3758724 A4 EP3758724 A4 EP 3758724A4
Authority
EP
European Patent Office
Prior art keywords
aav
vectors
associated virus
uses therefor
novel adeno
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP19760264.2A
Other languages
German (de)
English (en)
Other versions
EP3758724A1 (fr
Inventor
James M. Wilson
April TEPE
Kevin Turner
Joshua Joyner SIMS
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Pennsylvania Penn
Original Assignee
University of Pennsylvania Penn
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Publication of EP3758724A1 publication Critical patent/EP3758724A1/fr
Publication of EP3758724A4 publication Critical patent/EP3758724A4/fr
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
EP19760264.2A 2018-02-27 2019-02-27 Nouveaux vecteurs de virus adéno-associés (vaa), vecteurs de vaa présentant une désamidation de capside réduite et utilisations associées Pending EP3758724A4 (fr)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201862635964P 2018-02-27 2018-02-27
US201862667585P 2018-05-29 2018-05-29
US201862677471P 2018-05-29 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US201862722382P 2018-08-24 2018-08-24
PCT/US2019/019804 WO2019168961A1 (fr) 2018-02-27 2019-02-27 Nouveaux vecteurs de virus adéno-associés (vaa), vecteurs de vaa présentant une désamidation de capside réduite et utilisations associées

Publications (2)

Publication Number Publication Date
EP3758724A1 EP3758724A1 (fr) 2021-01-06
EP3758724A4 true EP3758724A4 (fr) 2022-07-06

Family

ID=67805569

Family Applications (1)

Application Number Title Priority Date Filing Date
EP19760264.2A Pending EP3758724A4 (fr) 2018-02-27 2019-02-27 Nouveaux vecteurs de virus adéno-associés (vaa), vecteurs de vaa présentant une désamidation de capside réduite et utilisations associées

Country Status (13)

Country Link
US (1) US20200407750A1 (fr)
EP (1) EP3758724A4 (fr)
JP (3) JP2021516046A (fr)
KR (1) KR20210010434A (fr)
CN (1) CN112352050A (fr)
AU (1) AU2019227726A1 (fr)
BR (1) BR112020017348A2 (fr)
CA (1) CA3091806A1 (fr)
CL (2) CL2020002200A1 (fr)
IL (1) IL276859A (fr)
MX (1) MX2020008933A (fr)
SG (1) SG11202008182TA (fr)
WO (1) WO2019168961A1 (fr)

Families Citing this family (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3356390T3 (pl) 2015-09-28 2021-07-05 The University Of North Carolina At Chapel Hill Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2021067598A1 (fr) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Procédés pour une utilisation thérapeutique améliorée d'aav recombinant
CA3157700A1 (fr) 2019-10-17 2021-04-22 Stridebio, Inc. Vecteurs de virus adeno-associes pour le traitement de la maladie de niemann-pick de type c
CA3155016A1 (fr) * 2019-10-21 2021-04-29 James M. Wilson Variants d'aav3b presentant un rendement de production et un tropisme hepatique ameliores
CA3165911A1 (fr) * 2020-01-03 2021-07-08 Sarepta Therapeutics, Inc. Procedes d'analyse de proteines de capside vaa
BR112022015921A2 (pt) * 2020-02-14 2022-10-04 Ultragenyx Pharmaceutical Inc Terapia gênica para tratar o transtorno de deficiência de cdkl5
EP4127189A1 (fr) 2020-03-31 2023-02-08 Ultragenyx Pharmaceutical Inc. Thérapie génique pour le traitement de l'acidémie propionique
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
US20230270884A1 (en) 2020-07-13 2023-08-31 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
AU2021359874A1 (en) * 2020-10-18 2023-05-25 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
EP4236975A1 (fr) * 2020-10-29 2023-09-06 The Trustees of The University of Pennsylvania Capsides de vaa et compositions les contenant
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
US20240091380A1 (en) 2021-02-01 2024-03-21 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
EP4323520A1 (fr) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Compositions utiles pour le traitement de l'amyotrophie spinale et bulbaire (sbma)
WO2022226263A1 (fr) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Nouvelles compositions présentant des motifs de ciblage spécifiques au cerveau et compositions les contenant
CN116444626A (zh) * 2021-12-28 2023-07-18 成都弘基生物科技有限公司 经过修饰的aav衣壳蛋白及其用途
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
WO2023147304A1 (fr) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Capsides d'aav pour une transduction cardiaque améliorée et un ciblage du foie
WO2023172491A1 (fr) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. Systèmes et procédés de production d'aav par lots modifiés
WO2023196892A1 (fr) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Immunisation passive avec des anticorps neutralisants anti-aav pour empêcher la transduction hors cible de vecteurs aav administrés par voie intrathécale
CN115011601B (zh) * 2022-06-27 2023-07-21 山东大学齐鲁医院 一种干扰JUND表达的shRNA、重组腺相关病毒载体及其应用
WO2024015966A2 (fr) * 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Vaa recombinants ayant des capsides de clade d et de clade e de vaa et compositions les contenant

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017100674A1 (fr) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif pour virus adéno-associé (aav) 1
WO2017180854A1 (fr) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Nouvelles capsides mutantes de aav8 et compositions les contenant
WO2018035059A1 (fr) * 2016-08-15 2018-02-22 Genzyme Corporation Méthodes de détection d'aav

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2648241T3 (es) * 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
PT2839014T (pt) * 2012-04-18 2021-03-19 Childrens Hospital Philadelphia ¿composição e métodos para transferência de genes altamente eficiente com a utilização de variantes de capsídeo de aav
WO2015013313A2 (fr) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Compositions et variants de virus adéno-associés, et méthodes et utilisations pour un transfert de gènes dans des cellules, des organes et des tissus
RS65241B1 (sr) * 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017100674A1 (fr) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif pour virus adéno-associé (aav) 1
WO2017180854A1 (fr) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Nouvelles capsides mutantes de aav8 et compositions les contenant
WO2018035059A1 (fr) * 2016-08-15 2018-02-22 Genzyme Corporation Méthodes de détection d'aav

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
APRIL R. GILES ET AL: "Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function", MOLECULAR THERAPY, vol. 26, no. 12, 1 December 2018 (2018-12-01), US, pages 2848 - 2862, XP055635211, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2018.09.013 *
APRIL ROSE GILES ET AL: "IMMUNOLOGICAL AND BIOCHEMICAL EVALUATION OF THE AAV CAPSID TO ADVANCE NEXT-GENERATION GENE THERAPY VECTOR DESIGN of Microbiology", 2018, pages 1 - 159, XP055757209, Retrieved from the Internet <URL:chttps://www.proquest.com/docview/2117235320/343F6ED3BA0A49C5PQ/7?accountid=29404> [retrieved on 20201207] *
GILES APRIL R ET AL: "223. The Biochemical and Functional Effects of Spontaneous Deamidation of the AAV Capsid on Gene Therapy Vectors", MOLECULAR THERAPY, vol. 22, no. SUPPL. 1, 1 May 2014 (2014-05-01), pages S85, XP055857502, Retrieved from the Internet <URL:https://www.cell.com/action/showPdf?pii=S1525-0016(16)35236-4> *
GILES APRIL R ET AL: "Supplemental Information: Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function", 1 December 2018 (2018-12-01), pages 1 - 13, XP055921625, Retrieved from the Internet <URL:https://ars.els-cdn.com/content/image/1-s2.0-S1525001618304544-mmc1.pdf> [retrieved on 20220516] *

Also Published As

Publication number Publication date
WO2019168961A1 (fr) 2019-09-06
MX2020008933A (es) 2021-01-15
SG11202008182TA (en) 2020-09-29
CL2022003757A1 (es) 2023-05-19
JP2023159235A (ja) 2023-10-31
CL2020002200A1 (es) 2021-01-29
JP2024041967A (ja) 2024-03-27
IL276859A (en) 2020-10-29
EP3758724A1 (fr) 2021-01-06
US20200407750A1 (en) 2020-12-31
JP2021516046A (ja) 2021-07-01
AU2019227726A1 (en) 2020-09-10
CA3091806A1 (fr) 2019-09-06
CN112352050A (zh) 2021-02-09
BR112020017348A2 (pt) 2020-12-29
KR20210010434A (ko) 2021-01-27

Similar Documents

Publication Publication Date Title
EP3758724A4 (fr) Nouveaux vecteurs de virus adéno-associés (vaa), vecteurs de vaa présentant une désamidation de capside réduite et utilisations associées
EP3768695A4 (fr) Nouveaux vecteurs de virus adéno-associés (aav), vecteurs aav ayant une déamidation de capside réduite et leurs utilisations
IL286911A (en) Recombinant adeno-associated viruses and their uses
ZA201905407B (en) Adeno-associated virus (aav) clade f vector and uses therefor
IL286464A (en) A virus of vectors related to recombinant DNA
EP3890786A4 (fr) Vecteur viral adéno-associé recombiné pour l&#39;insertion de gènes
WO2018022608A3 (fr) Nouvelles protéines de capside de virus adéno-associés
EP3645021A4 (fr) Vecteurs viraux adéno-associés destinés à la thérapie génique
EP3943503A4 (fr) Virus adéno-associé à variant de protéine capsidique et utilisation associée
EP3575398A4 (fr) Mutant de protéine de capside de virus adéno-associé (vaa)
IL282885A (en) AAV viral vectors and their uses
EP3947700A4 (fr) Virus adéno-associé comprenant une capside modifiée
EP4303225A3 (fr) Vecteurs viraux adéno-associés polyploïdes et leurs procédés de fabrication et d&#39;utilisation
CA2921232A1 (fr) Vecteurs du facteur viii de virus associe aux adenovirus
EP4041755A4 (fr) Capsides de vaa modifiées et leurs utilisations
MX2021012564A (es) Formulaciones y métodos de vectores de virus adenoasociados.
EP3947422A4 (fr) Vecteurs de virus adéno-associés (aav) modifiés pour l&#39;expression transgénique
EP3807297A4 (fr) Capsides synthétiques de virus adéno-associé du foie et leurs utilisations
EP3891290A4 (fr) Lignée cellulaire productrice de virus adéno-associé (aav) et procédés associés
EP3790979A4 (fr) Administration de virus adéno-associé (aav) d&#39;anticorps anti-fam19a5
EP3959324A4 (fr) Vecteurs aav codant pour mini-pcdh15 et leurs utilisations
EP4048794A4 (fr) Vecteurs de virus adéno-associés à triple fonction (aav) pour le traitement de maladies associées à c9orf72
EP3956439A4 (fr) Virus de la peste porcine classique recombiné
EP3746560A4 (fr) Série prête à la campagne de plasmides de complémentation de virus recombinant adéno-associé (raav)
EP3655041A4 (fr) Vecteurs adéno-associés recombinants

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20200924

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

AX Request for extension of the european patent

Extension state: BA ME

RIN1 Information on inventor provided before grant (corrected)

Inventor name: SIMS, JOSHUA, JOYNER

Inventor name: TEPE, APRIL

Inventor name: WILSON, JAMES, M.

Inventor name: TURNER, KEVIN

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40044078

Country of ref document: HK

RIC1 Information provided on ipc code assigned before grant

Ipc: C12N 15/861 20060101ALI20220131BHEP

Ipc: C12N 15/86 20060101ALI20220131BHEP

Ipc: C12N 15/09 20060101ALI20220131BHEP

Ipc: C12N 7/00 20060101ALI20220131BHEP

Ipc: A61K 35/761 20150101ALI20220131BHEP

Ipc: A61K 35/76 20150101AFI20220131BHEP

A4 Supplementary search report drawn up and despatched

Effective date: 20220603

RIC1 Information provided on ipc code assigned before grant

Ipc: C12N 15/861 20060101ALI20220530BHEP

Ipc: C12N 15/86 20060101ALI20220530BHEP

Ipc: C12N 15/09 20060101ALI20220530BHEP

Ipc: C12N 7/00 20060101ALI20220530BHEP

Ipc: A61K 35/761 20150101ALI20220530BHEP

Ipc: A61K 35/76 20150101AFI20220530BHEP

P01 Opt-out of the competence of the unified patent court (upc) registered

Effective date: 20230706